Genes associated with “FXTAS

56 genes foundOpen Targets: fragile X-associated tremor/ataxia syndrome
How are genes scored? (0–100 composite)
-5–15
ClinGen
0–15
HPO Freq
0–15
Open Targets
0–12
Phen2Gene
0–10
ClinVar
0–8
Constraint
0–8
Dosage
0–8
OMIM CS
0–8
PanelApp
0–5
OMIM
0–5
G2P
0–4
Breadth
Tiers:Strong Candidates (≥20)Consider (≥8)Possible (≥3)
Evidence dots:
HPOClinVarPhen2GeneOpen TargetsPanelApp
hover for details

Strong Candidates

1 gene
1
FMR1

fragile X messenger ribonucleoprotein 1

30
score
ClinGen: DefinitiveGTR ↑

Fragile X syndrome

Frequency
-
P/LP Variants
18
OT Score
0.70

Consider

4 genes

FRAGILE X TREMOR/ATAXIA SYNDROME; FXTAS

9PURA
Def

Neurodevelopmental disorder with neonatal respiratory insufficiency, hypotonia, and feeding difficulties

8GABRA1
Def

gamma-aminobutyric acid type A receptor subunit alpha1

?Inclusion body myopathy with early-onset Paget disease with or without frontotemporal dementia 2

Possible

22 genes — click to expand

Neuronal intranuclear inclusion disease

6GABRB1
Lim

gamma-aminobutyric acid type A receptor subunit beta1

CUGBP- AND ELAV-LIKE FAMILY, MEMBER 1; CELF1

6PMP22
Def

peripheral myelin protein 22

glutamate metabotropic receptor 1

6HPRT1
Def

hypoxanthine phosphoribosyltransferase 1

gamma-aminobutyric acid type A receptor subunit alpha5

4GABRB2
Def

gamma-aminobutyric acid type A receptor subunit beta2

4GABRB3
Def

gamma-aminobutyric acid type A receptor subunit beta3

4GABRG2
Def

gamma-aminobutyric acid type A receptor subunit gamma2

4ATP2B2
Def

ATPase plasma membrane Ca2+ transporting 2

4KCNMA1
Def

potassium calcium-activated channel subfamily M alpha 1

4SPTBN4
Def

spectrin beta, non-erythrocytic 4

4PSAP
Def

prosaposin

4NPC1
Def

NPC intracellular cholesterol transporter 1

4SCN8A
Def

sodium voltage-gated channel alpha subunit 8

4RELN
Def

reelin

4DDC
Def

dopa decarboxylase

3GABRD
Mod

gamma-aminobutyric acid type A receptor subunit delta

Data aggregated from HPO/Monarch, ClinVar, OMIM (phenotypes + clinical synopsis), Open Targets, PanelApp, Phen2Gene, and Gene2Phenotype. Click a gene to view full details.